Recent Articles

An informative article on the recent and tragic death of a young child in a gene therapy trial for Rett syndrome.

"Like other parents of children with Rett syndrome, Coenraads wishes scientists would hurry up and develop treatments. But as the founder and CEO of the Rett Syndrome Research Trust, she’s in a position to do something about it.”

Neurogene announced that NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for the treatment of Rett syndrome. The designation was based on preliminary clinical evidence from the ongoing Phase 1/2 clinical trial with NGN-401 that shows potential to ...

Taysha reports financial results as well as updates on their pediatric and adult clinical trials.